Mitapivat Granted Priority Review for Pyruvate Kinase Deficiency
Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes.
Anemias
MPR Weekly Dose Podcast Episode 89
Big news on the COVID-19 vaccine booster dose. The CDC issues new recommendations on vaccines. What protection the Moderna vaccine offers after 6 months. A treatment approved for Pompe disease. And the FDA denies an application for a new roxadustat indication.
FDA Denies Approval of Roxadustat for Anemia in CKD
The FDA noted that the application could not be approved in its present form and had requested an additional clinical study be conducted.
Generic Version of Feraheme Now Available
The first generic version of Feraheme (ferumoxytol injection) has been made available by Sandoz.
FDA Panel Votes on Roxadustat for Anemia in Chronic Kidney Disease
Concerns over thromboembolic events and MACE risk were discussed during the meeting.
Treatment at Infusion Center Beneficial in Sickle Cell Disease
Outcomes significantly better for treatment of vaso-occlusive crises at infusion center versus emergency department
Renoprotective Effect of SGLT2 Inhibitors Mediated Largely by Hematocrit
Increases in the proportion of red blood cells mediates the reduction in renal risks associated with SGLT2 inhibitor therapy in patients with type 2 diabetes and cardiovascular disease, a study found.
Ultomiris Approved for Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria
The approval was based on data from an open-label, phase 3 study that assessed Ultomiris in 13 patients 9 through 17 years of age with PNH.
Empaveli Approved for Paroxysmal Nocturnal Hemoglobinuria
Empaveli is a complement inhibitor that binds to complement protein C3 and its activation fragment C3b.
FDA Approves Single Dose Option for Iron Replacement Therapy Injectafer
For patients weighing at least 50kg, Injectafer may be administered as a single dose option of 15mg/kg body weight up to a maximum of 1000mg intravenously.
